WORLDSymposium

The president of MAGIC Clinic and CEO of Discovery DNA discussed the implications of gene therapy data presented at the 2025 WORLDSymposium.

Reena Sharma, MD, an adult metabolic consultant at Salford Royal Hospital, discussed areas of interest for future study for the Gaucher disease gene therapy product.

The adult metabolic consultant at Salford Royal Hospital discussed areas of interest for future study for the Gaucher disease gene therapy product.

Reena Sharma, MD, an adult metabolic consultant at Salford Royal Hospital, discussed results she presented at the 21st Annual WORLDSymposium.

The adult metabolic consultant at Salford Royal Hospital discussed results she presented at the 21st Annual WORLDSymposium.

A positive mean eGFR slope was seen in 23 patients who reached 1 year of follow-up in the phase 1/2 STAAR trial.

With regard to safety, Ultragenyx characterized the gene therapy as “generally well-tolerated."

Among the 5 patients in the efficacy set, 4 patients ceased receiving their previous standard of care therapy.

Paul Harmatz, MD, professor, UCSF, discussed benefits in neurocognitive outcomes and ERT therapy usage after RGX-121 gene therapy for Hunter syndrome.

The associate professor of clinical pediatrics at Cincinnati Children's discussed the latest data from the phase 1/2 STAAR study of isaralgagene civaparvovec.

Investigators concluded that the data support the use of HS as a predictive biomarker in Sanfilippo syndrome.

The executive director of global clinical development at Ultragenyx Pharmaceutical discussed the statistical findings she presented at the 2024 WORLDSymposium.

The executive director of global clinical development at Ultragenyx Pharmaceutical discussed UX111, the company’s investigational gene therapy for MPSIIIA.

The gene-edited cell therapy also showed a safety profile consistent with that previously seen in patients with early-onset MLD.

Data from defunct company Lysogene’s discontinued trial of LYS-GM101 were presented at WORLDSymposium.

Among the 5 patients treated in the study, sustained supraphysiological expression of the disease-targeted gene, SGSH, was rapidly achieved in the leukocytes and the plasma.

Hematological recovery was achieved and maintained in all 4 patients in the study.

Orchard Therapeutics has also randomized the first patient with Hurler Syndrome in the phase 3, registrational HURCULES trial of OTL-203 gene therapy.

The associate professor of clinical pediatrics at Cincinnati Children's Hospital Medical Center discussed Sangamo Therapeutics’ gene therapy, ST-920, and the phase 2 STAAR clinical trial.

Catch up with some of CGTLive’s top news articles from the annual WORLDSymposium.

The associate professor of clinical pediatrics at Cincinnati Children's Hospital Medical Center discussed the limitations of the current standard of care for Fabry disease.

Samiah Al-Zaidy, MD, vice president of clinical development and lead on the GM1 Program at Passage Bio, discussed the latest results from the Imagine-01 clinical trial evaluating the investigational treatment PBGM01.

Four of 5 patients showed improvements in ejection fraction following bone marrow transplant.

4D Molecular Therapeutics’ 4D-310 effected clinically meaningful improvements in left ventricular function, exercise capacity, and quality of life.

All dose-escalation patients withdrew from enzyme replacement therapy (ERT) and remain off ERT.

Ultragenyx’s self-complementary AAV9-based gene therapy reduced levels of relevant CSF biomarkers, as well as improvements in neurocognitive assessments and behavioral domains, among patients without neurodegeneration.

Patients who received the high dose of PBGM01 showed an increase in β-Gal activity in the CSF of 4.7 to 5.2 times baseline.

Most treated participants were within 2 SDs of normative mean in acquiring cognition, expressive language and fine motor skills.

Interim data from the phase 1/2 CAMPSIITE trial were presented at WORLDSymposium 2023.

Al-Zaidy, the vice president of clinical development and lead on the GM1 Program at Passage Bio, discussed the results of the phase 1/2 Imagine-01 clinical trial of PBGM01.